Abstract
Drug development for rare and intractable diseases has been challenging for decades due to the low prevalence and insufficient information on these diseases. Drug repositioning is increasingly being used as a promising option in drug development. We aimed to analyze the trend of drug repositioning and inter-disease drug repositionability among rare and intractable diseases. We created a list of rare and intractable diseases based on the designated diseases in Japan. Drug information extracted from clinical trial data were integrated with information of drug target genes, which represent the mechanism of drug action. We obtained 753 drugs and 551 drug target genes from 8307 clinical trials for 189 diseases or disease groups. Trend analysis of drug sharing between a disease pair revealed that 1676 drug repositioning events occurred in 4401 disease pairs. A score, Rgene, was invented to investigate the proportion of drug target genes shared between a disease pair. Annual changes of Rgene corresponded to the trend of drug repositioning and predicted drug repositioning events occurring within a year or two. Drug target gene-based analyses well visualized the drug repositioning landscape. This approach facilitates drug development for rare and intractable diseases.
Highlights
Drug development for rare and intractable diseases has been challenging for decades due to the low prevalence and insufficient information on these diseases
Comprehensive information on drug development is available from clinical trial registries, since it is mandatory to register all clinical trials in at least one registry worldwide
In order to search clinical trials, we constructed an original list of rare and intractable diseases based on the intractable diseases designated in Japan (Supplementary Table 1)
Summary
Drug development for rare and intractable diseases has been challenging for decades due to the low prevalence and insufficient information on these diseases. Drug target gene-based analyses well visualized the drug repositioning landscape This approach facilitates drug development for rare and intractable diseases. Drug development in rare and intractable diseases has attracted the attention of pharmaceutical companies. New modalities, such as antibodies and anti-sense oligonucleotides, are being developed as an effective approach to these diseases. When repositioning a drug developed for a specific disease, it is important to find a target disease that has a common mechanism of action for the particular d rug[6,7,8]. Sharing the target genes of a drug between a disease pair may suggest that this disease pair shares a common mechanism of drug action. A disease pair sharing drug target genes could be a good candidate for drug repositioning
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